Protocols

Jakobovits abruptly exits Adicet; Stanford researchers uncover mechanistic details of ALS using CRISPR

Aya Jakobovits

→ Aya Jakobovits has abruptly left the top position at cell therapy player Adicet. Jakobovits, the first CEO at Kite, is staying on the board of the Menlo Park, CA biotech and will continue as an adviser to the company. Donald Santel is stepping up as interim CEO while the company hunts for a successor.

→ Scientists at Stanford University School of Medicine say they’ve uncovered details about how amytrophic lateral sclerosis (ALS) progresses by using CRISPR-Cas9. The researchers applied the tech to sort through the human genome, picking up a deeper mechanistic understanding of the disease and identifying a handful of genes that could hold potential as drug targets, Stanford said in statement. ALS, which erodes muscle function and impairs the brain’s ability to communicate with the body, is a neurodegenerative disease. A signature of the condition is the buildup of abnormal protein clumps in the brain, which are toxic to neurons. “These toxic protein aggregates are what’s likely driving the pathology in the disease, but no one really knows how they cause neuronal cell death,” said Aaron Gitler, professor of genetics and co-author of the study. “That’s really what we wanted to probe in this study.” A paper describing the research was published in Nature Genetics.

→ Sanofi’s newly acquired Bioverativ has dosed the first patient in pivotal program for BIVV009 for cold agglutinin disease (CAgD). The Phase III program includes two parallel trials, Cardinal and Cadenza. There are no approved therapies for the disease.


With additional reporting by Brittany Meiling.

 


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